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Thalidomide for the Treatment of Primary Sclerosing Cholangitis (PSC)

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ClinicalTrials.gov Identifier: NCT00953615
Recruitment Status : Terminated (Lack of enrollment)
First Posted : August 6, 2009
Results First Posted : February 27, 2012
Last Update Posted : February 27, 2012
Sponsor:
Collaborator:
Celgene Corporation
Information provided by:
Mayo Clinic

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Primary Sclerosing Cholangitis
Intervention Drug: Thalidomide
Enrollment 1
Recruitment Details Participants were recruited between 4/6/2006 and 5/7/2009 at Mayo Clinic outpatient area.
Pre-assignment Details  
Arm/Group Title Thalidomide
Hide Arm/Group Description Patients with primary sclerosing cholangitis (PSC) who met with enrollment criteria were treated with 400 mg in the evening for a duration of 6 months
Period Title: Overall Study
Started 1
Completed 0
Not Completed 1
Reason Not Completed
Adverse Event             1
Arm/Group Title Thalidomide
Hide Arm/Group Description Patients with primary sclerosing cholangitis (PSC) who met with enrollment criteria were treated with 400 mg in the evening for a duration of 6 months
Overall Number of Baseline Participants 1
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
<=18 years
0
   0.0%
Between 18 and 65 years
1
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
Female
0
   0.0%
Male
1
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 1 participants
1
1.Primary Outcome
Title Alkaline Phosphatase, Aspartate Aminotransferase, Alanine Aminotransferase
Hide Description The primary outcome was the change in serum liver biochemical parameter levels after 6 months of thalidomide when compared to baseline values. This was to be analyzed using the nonparametric Wilcoxon signed rank test of significance. This was based on the non-normal distribution of serum hepatic biochemical parameters among patients with PSC and the continuous nature of these variables.
Time Frame 6 months, baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was not performed because participant did not complete the study due to adverse events.
Arm/Group Title Thalidomide
Hide Arm/Group Description:
Only one subject was enrolled, so study was terminated early.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Overall Toxicity and Tolerability
Hide Description Overall toxicity and tolerability were to be measured by the number of patients with development of neuropathy, increased liver biochemistries, drowsiness, dizziness and orthostatic hypotension.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Thalidomide
Hide Arm/Group Description:
Only one subject was enrolled, so study was terminated early.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Mayo Risk Score
Hide Description The Mayo Risk Score estimates the survival probability of a patient with primary sclerosing cholangitis based on the following variables: age, bilirubin, albumin, AST and history of variceal bleeding.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Thalidomide
Hide Arm/Group Description:
Only one subject was enrolled, so study was terminated early.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Soluble Tumor Necrosis Factor - Alpha
Hide Description Assessment of effect from thalidomide on soluble tumor necrosis factor – alpha compared to baseline values were to be performed at study conclusion.
Time Frame 6 months, baseline
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Thalidomide
Hide Arm/Group Description:
Only one subject was enrolled, so study was terminated early.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 28 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Thalidomide
Hide Arm/Group Description Patients with primary sclerosing cholangitis (PSC) who met with enrollment criteria were treated with 400 mg in the evening for a duration of 6 months
All-Cause Mortality
Thalidomide
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Thalidomide
Affected / at Risk (%) # Events
Total   0/1 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Thalidomide
Affected / at Risk (%) # Events
Total   1/1 (100.00%)    
Cardiac disorders   
low blood pressure  1  1/1 (100.00%)  1
Gastrointestinal disorders   
loose stools  1  1/1 (100.00%)  1
decreased appetite  1  1/1 (100.00%)  1
aphthous stomatitis  1  1/1 (100.00%)  1
General disorders   
fatigue  1  1/1 (100.00%)  1
weight loss  1  1/1 (100.00%)  1
Nervous system disorders   
hand pain  1  1/1 (100.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Only one subject was enrolled, so study was terminated.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Keith D. Lindor, M.D.
Organization: Mayo Clinic
Phone: 507-284-2969
Responsible Party: Dr. K. Lindor, Mayo Clinic
ClinicalTrials.gov Identifier: NCT00953615     History of Changes
Other Study ID Numbers: 342-06
First Submitted: August 4, 2009
First Posted: August 6, 2009
Results First Submitted: August 8, 2011
Results First Posted: February 27, 2012
Last Update Posted: February 27, 2012